Phase II Study of Treatment for Newly Diagnosed Multiple Myeloma Patients Over 75 Years Old with Alternating Bortezomib/dexamethasone and Lenalidomide/dexamethasone: the MARBLE Trial

Elderly multiple myeloma (MM) patients, who are generally ineligible for transplantation, have high risks of death and treatment discontinuation, and require a regimen incorporating novel agents that balance safety, tolerability, and efficacy. We evaluated alternating bortezomib-dexamethasone and lenalidomide-dexamethasone treatments administered over a 63-day cycle in transplant-ineligible elderly patients with newly diagnosed MM. Subcutaneous bortezomib 1.3 mg/m2 was administered weekly on Days 1, 8, 15, and 22; oral lenalidomide 15 mg daily on Days 36-56; and oral dexamethasone 20 mg on Days 1, 8, 15, 22, 36, 43, 50, and 57 for 6 cycles. The primary endpoint was the overall response rate

Phase II Trial Using Romidepsin after Gemcitabine, Dexamethasone, and Cisplatin Therapy in Elderly Transplant-Ineligible Patients with Relapsed/Refractory Peripheral T-Cell Lymphoma: Study Protocol

Romidepsin is an important therapeutic option for patients with peripheral T-cell lymphoma (PTCL). However, the timing of romidepsin administration remains controversial. Romidepsin was launched in Japan as a consolidation therapy agent after conventional salvage chemotherapy with gemcitabine, dexamethasone, and cisplatin (GDP). GDP therapy will be administered every 3 weeks. If complete response, partial response, or stable disease is confirmed after 2-4 GDP cycles, romidepsin will be administered every 4 weeks. The primary endpoint is a 2-year progression-free survival rate. Patients participating in this study and undergoing treatment can expect results similar to or better than those of conventional therapies

A Phase I/II Study of Crizotinib for Recurrent or Refractory Anaplastic Lymphoma Kinase-Positive Anaplastic Large Cell Lymphoma and a Phase I Study of Crizotinib for Recurrent or Refractory Neuroblastoma : Study Protocol for a Multicenter Single-arm Open-label Trial

Crizotinib is an inhibitor of multiple tyrosine kinases, including the anaplastic lymphoma kinase (ALK). Responses to crizotinib have also been reported in patients with ALK-positive anaplastic large-cell lymphoma (ALCL) and solid tumors with ALK-mutation, including neuroblastoma. Optimal treatment for patients with recurrent or refractory ALK-positive ALCL and neuroblastoma has not been established. There is a need to develop new drugs for these patients. The objectives of this trial are to evaluate the tolerability and safety of crizotinib in Japanese patients with recurrent/refractory ALK-positive ALCL or neuroblastoma (phase I) and its efficacy in recurrent/refractory ALK-positive ALCL (phase II)

A Multicenter, Open-label, Clinical Trial to Assess the Effectiveness and Safety of Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced-intensity Conditioning in Relapsed/refractory Anaplastic Large-cell Lymphoma in Children

No standard treatment for relapsed or refractory anaplastic large-cell lymphoma (ALCL) has been established. This study is a multicenter, open-label trial to examine the effectiveness and safety of transplantation with reduced-intensity conditioning (RIC) for patients under 20 years old with relapsed or refractory ALCL. We defined RIC as the administration of fludarabine (30 mg/m2/day) for five days plus melphalan (70 mg/m2/day) for two days and total body irradiation at 4 Gy, followed by allogeneic hematopoietic stem cell transplantation

High-dose Dexamethasone Therapy as the Initial Treatment for Idiopathic Thrombocytopenic Purpura: Protocol for a Multicenter, Open-label, Single Arm Trial

Standard therapy for idiopathic thrombocytopenic purpura (ITP) has not been established. We are conducting a multicenter, prospective trial to determine the efficacy and safety of short-term, high-dose dexamethasone therapy in ITP patients aged 18-80 years with platelet counts of <20, 000 /μL, or with <50, 000/ μL and bleeding symptoms. The primary endpoints of this trial are the proportion of responses (complete plus partial response) on day 180 (day 46+180) after the completion of the 46-day high-dose dexamethasone therapy. The results of this investigation of the effectiveness and safety of this regimen will be essential for the establishment of standard therapy for ITP

R-mini CHP in ≥80-year-old Patients with Diffuse Large B-cell Lymphoma: A Multicenter, Open-label, Single-arm Phase II Trial Protocol

In very-elderly diffuse large B-cell lymphoma (DLBCL) patients, treatment intensities must be lowered due to the risks of comorbidities and organ function deterioration, and treatment outcomes are worse compared to younger patients. Very-elderly patients are often excluded from DLBCL clinical trials, and optimal treatments and dosages are not established. In this clinical trial, we examined the efficacy and safety of 6 courses of R-mini CHP therapy (cf., CHOP [cyclophosphamide, doxorubicin, vincristine, and prednisone]) in which vincristine is omitted to avoid the peripheral neuropathy that reduces elderly patients’ quality of life, as remission induction therapy in DLBCL patients aged≥80 years

Phase II Study of Pleurodesis using Sterile Graded Talc in Patients with Secondary Intractable Pneumothorax: Protocol for a Multicentre, Open-label Single-arm Trial

A pneumothorax can be primary or secondary. A high proportion of patients with secondary spontaneous pneumothorax are the elderly who are in poor general condition due to their impaired cardiac and pulmonary functions as well as due to other complications. Therefore, it may be difficult for these patients to undergo surgical procedures; in addition, the elderly may be at high risk for postoperative pulmonary fistula due to severe adhesions and emphysema complications. These non-operative and high-risk cases may be treated with pleurodesis (a procedure that involves instillation of a chemical or irritant into the thoracic cavity through an injection), bronchoscopic bronchial embolisation, or other procedures. In Japan, no device is currently approved for performing pleurodesis, but an approval of one device is expected soon. This will be an open-label, single-arm multicentre study conducted among 30 patients with secondary intractable pneumothorax who are not indicated to undergo surgery. The primary endpoint will be presence or absence of chest tube removal. The secondary endpoints will be the disappearance/decrease of air leakage, grade of dyspnoea, and duration of drainage. This study will assess the safety and efficacy of sterile graded talc pleurodesis in patients with secondary intractable pneumothorax.This research is (partially) supported by the Project Promoting Clinical Trials for Development of New Drugs and Medical Devices (Japan Medical Association) and Early/Exploratory Clinical Trial Center Development Projects from the Japan Agency for Medical Research and Development (AMED). This study is registered in the Center for Clinical Trials, Japan Medical Association (JMA-IIA00272)

An Open-label Single-arm Trial of a Novel Extramedullary Guide Coordinated with 3D Surgical Assistive Software for Total Knee Arthroplasty

There is no assistive device for extramedullary surgery coordinated with 3D surgical assistive software for the total knee arthroplasty (TKA). We developed a novel extramedullary universal guide coordinated with 3D surgical assistive software and a novel extramedullary patient-specific assistive guide for the placement of femoral components by referring to an area not affected by cartilage or bone spurs, and filed a patent application. In this study, we visualize and reconstruct the total alignment of the lower extremity in TKA using these surgical devices, and validate their precision. A report releasing study results will be submitted in an appropriate journal

Development of an Oropharyngeal Scope with an Integrated Tongue Depressor: NTOP2013 Study

The oropharynx is examined with a light source such as an electric light, a penlight, or a forehead mirror based on an acquired visual field using a tongue depressor. However, it is extremely difficult to obtain objective and reproducible images of tissue within the pharynx required in recent years with these methods, and insufficient progress in the examination tools has been made. There is an increasing need to develop a method for display during oropharyngeal examination. We conducted the present study to develop a novel oropharyngeal endoscope as an objective observation method

Rationale and Design of a Prospective, Multicentre, Stop Tyrosine Kinase Inhibitor Trial of Paediatric Patients with Chronic Myeloid Leukaemia with Sustained Complete Molecular Response (STKI-14)

Chronic myeloid leukaemia (CML) is a relatively rare disease in children, accounting for 2–3% of all paediatric leukaemia cases. Generally, children with CML can avoid hematopoietic stem cell transplantation and achieve molecular responses with tyrosine kinase inhibitors (TKI). However, CML stem cells are thought to survive in many patients, even after TKI treatment. Many aspects of the toxic effects of prolonged exposure to TKIs during childhood remain unclear, particularly those regarding growth impairment. This lack of clarity underscores the importance of the present clinical trial, which aims to clarify the feasibility of treatment-free remission (TFR) in children following TKI treatment. We aim to examine the long-term out-comes and complications of TKIs before and after cessation to better understand the unknown complications that could arise in adulthood. This trial targets patients who were diagnosed with CML at an age younger than 20 years, were in the chronic or accelerated phase at initial diagnosis and remained in complete molecular remission for at least 2 years after TKI administration. We will examine the utility of TKI cessation and assess the treatment results of patients who resumed TKI therapy after losing a major molecular response. We will also investigate factors related to the feasibility of a TFR after TKI cessation